Entrada Therapeutics Announces Collaboration with the Myotonic Dystrophy Clinical Research Network to Study the Natural History of Myotonic Dystrophy Type 1
The natural history study will generate data to further the ongoing efforts of the myotonic dystrophy research community to understand disease progression and identify potential clinical outcome measures and endpoints for clinical trials
Entrada recently announced new preclinical data that supports further development of the Company’s second clinical candidate, ENTR-701, for the potential treatment of myotonic dystrophy type 1
BOSTON, Aug. 04, 2022 (GLOBE NEWSWIRE) -- Entrada Therapeutics, Inc. (Nasdaq: TRDA), a biopharmaceutical company aiming to transform the lives of patients by establishing intracellular Endosomal Escape Vehicle (EEV™) therapeutics as a new class of medicines, today announced a new collaboration with the Myotonic Dystrophy Clinical Research Network (DMCRN) supporting END-DM1 (Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1). END-DM1 is a natural history study to advance the understanding of disease progression in patients with myotonic dystrophy type 1 (DM1). This is a non-interventional study designed and conducted by the DMCRN, a network of medical centers with expertise in DM1 clinical care and research that is collecting data to support future clinical trials of potential therapies for DM1.
“Entrada is developing new therapeutic options that will potentially transform the treatment of devastating diseases and make a meaningful difference in the lives of patients living with myotonic dystrophy type 1, a disease where there are currently no approved therapies,” said Nerissa Kreher, MD, Chief Medical Officer of Entrada Therapeutics. “Partnering with the DM1 community through our support of the END-DM1 study will provide important data regarding biomarkers and clinical endpoints in DM1, informing the design of our future clinical trials and the ongoing development of potential DM1 treatments.”
Entrada’s Endosomal Escape Vehicle (EEV™)-conjugated phosphorodiamidate morpholino oligomer (PMO) is designed to enable the efficient intracellular delivery of a wide range of therapeutics, previously considered inaccessible and undruggable, into a variety of organs and tissues. Entrada’s EEV-PMO uses an allele-specific approach that sterically blocks the triplet repeats in the mRNA that sequesters these critical proteins in order to restore muscle function. At the TIDES USA 2022: Oligonucleotide & Peptide Therapeutics Conference, Entrada’s clinical candidate ENTR-701 for DM1 was supported by new preclinical data indicating prolonged splicing correction in the tibialis anterior, triceps and quadriceps, and amelioration of myotonia in a DM1 mouse model following a single dose.
“We are excited to have Entrada join the DMCRN collaboration and participate in our shared mission to transform the lives of people affected by neuromuscular diseases through the study of DM1,” said Nicholas E. Johnson, M.D., MSCI, FAAN, an associate professor, division chief of neuromuscular medicine and vice chair of research in the Department of Neurology at Virginia Commonwealth University’s School of Medicine, who serves as co-chair of the END-DM1 Study. “The data collected through the natural history study, END-DM1, and associated pilot studies, will provide critical knowledge for DM1 researchers and Entrada to successfully design DM1 clinical trials and potentially provide much needed treatment options for patients and families living with this serious condition.”
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